Fri Oct 07 2022

71 articles - From Friday Sep 30 2022 to Friday Oct 07 2022

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Guidelines

Guidelines, position statements, white papers, technical reviews, consensus statements, etc…

Am J Hematol

The International Consensus Classification of Myeloid Neoplasms and Acute Leukemias: Myeloproliferative Neoplasms.

This current in-depth review focuses on the ICC-2022 category of JAK2 mutation-prevalent myeloproliferative neoplasms (MPNs): essential thrombocythemia, polycythemia vera, primary myelofibrosis, and MPN, unclassifiable. The ICC MPN subcommittee chose to preserve the primary role of bone marrow morphology in disease classification and diagnostics, while also acknowledging the complementary role of genetic markers for establishing clonality, facilitating MPN subtype designation, and disease prognostication.

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Ann Oncol

Oncology Phase I Trial Design and Conduct: Time for a Change MDICT Guidelines 2022.

The phase I dose escalation trial should define the recommended dose range for later testing in randomized phase 2 trials, rather than a single recommended phase 2 dose, and consider scenarios where different populations may require different dosages. The adoption of these recommendation will improve dosage selection in early clinical trials of new anti-cancer treatments and ultimately, outcomes for patients.

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J Hematol Oncol

Evidence-based expert consensus on the management of primary central nervous system lymphoma in China.

Patients with suspected primary vitreoretinal lymphoma (PVRL) should be diagnosed by vitreous biopsy. PVRL or PCNSL patients with concurrent VRL can be treated with combined systemic and local therapy.

Pubmed   Journal   ReadQx   PMC


Meta-analysis

meta-analyses and systematic reviews

Blood Adv

Systematic Review of Outcome Reporting in Studies Addressing Sickle Cell Disease and Pregnancy.

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Haematologica

Association of FLT3-internal tandem duplication length with overall survival in acute myeloid leukemia: a systematic review and metaanalysis - Authors' reply.

Not available.

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Association of FLT3-ITD length with overall survival in acute myeloid leukemia: a systematic review and meta-analysis.

Not available.

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Comment on: Antibody response after vaccination against SARS-CoV-2 in adults with haematological malignancies: a systematic review and meta-analysis.

Not available.

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Original articles

RCT, clinical trials, retrospective studies, etc…

Am J Hematol

Role of serum free light chain assay for defining response and progression in immunoglobulin secretory multiple myeloma.

sFLC PD adversely affected the OS after Pe compared to PD with increasing monoclonal Ig only (HR=0.52, p=0.012). Our results support the inclusion of the sFLC assay for defining response and PD in Ig-MM.

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Sex differences in lymphoma incidence and mortality by subtype: a population-based study.

In conclusion, we demonstrate a significantly higher incidence and trend towards higher mortality in men for most lymphoma subtypes. Future studies with large patient material that include detailed clinicopathological prognostic factors are warranted to further delineate and explain sex differences in lymphoma survival to enable optimal management of lymphoma patients regardless of sex.

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Blood

Assembly of von Willebrand Factor Tubules with in Vivo Helical Parameters Requires A1 Domain Insertion.

We reconstituted VWF tubules from segments containing the A1 domain and discovered it to be inserted between helical turns of the tubule, altering helical parameters and explaining the increased robustness of tubule formation when A1 is present. The conclusion from this observation is that the A1 domain has a direct role in VWF assembly, along with its known activity in hemostasis post-secretion.

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GENETIC SUBGROUPS INFORM ON PATHOBIOLOGY IN ADULT AND PEDIATRIC BURKITT LYMPHOMA.

IC-BL and DGG-BL and their prototypical genetic features (ID3 and TP53) had significant associations with patient outcomes that were different among aBL and pBL cohorts. These findings highlight shared pathogenesis between aBL and pBL, and establish genetic subtypes within BL that serve to delineate tumors with distinct molecular features, providing a new framework for epidemiological, diagnostic, and therapeutic strategies.

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Murine Allogeneic CAR-T Cells Integrated Before or Early After Posttransplant Cyclophosphamide Exert Anti-Tumor Effects.

In comparison with infusion on day +5, CAR-T-cell infusion on day 0 demonstrated superior clinical efficacy associated with earlier CAR-T-cell expansion, higher phenotypic CAR-T-cell activation, less CD4+CD25+Foxp3+ CAR-T-cell recovery, and transcriptional changes suggesting increased activation of CD4+ CAR-T-cells and more cytotoxic CD8+ CAR-T-cells. This study provides mechanistic insight into PTCy's impact on graft-versus-tumor immunity and describes novel approaches to integrate CAR-T-cells and allo-HCT that may compensate for deficiencies of each individual approach.

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The proto-oncogene TCL1A deregulates cell cycle and genomic stability in CLL.

This adds to our concept of oncogenic TCL1A by targeting genome stability. Overall, we propose that TCL1A acts as a pleiotropic adapter molecule with a synergistic net effect of multiple hijacked pathways.

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CA Cancer J Clin

Breast Cancer Statistics, 2022.

Black women have the lowest 5-year relative survival of any racial/ethnic group for every molecular subtype and stage of disease (except stage I), with the largest Black-White gaps in absolute terms for hormone receptor-positive/human epidermal growth factor receptor 2-negative disease (88% vs 96%), hormone receptor-negative/human epidermal growth factor receptor 2-positive disease (78% vs 86%), and stage III disease (64% vs 77%). Progress against breast cancer mortality could be accelerated by mitigating racial disparities through increased access to high-quality screening and treatment via nationwide Medicaid expansion and partnerships between community stakeholders, advocacy organizations, and health systems.

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Haematologica

Age- and gender-matched controls needed for platelet-based biomarker studies.

Not available.

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Associations between socioeconomic status and bispecific LV20.19 CAR T cell therapy outcomes.

Not available.

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CD56brightCD16- natural killer cells as an important regulatory mechanism in chronic graftversus-host disease.

Moreover, this is the first paper to clearly establish that a CD56brightCD3-CD16-perforin- NKreg population associates with a lack of cGvHD and has several unique characteristics, including the suppression of helper T cell function in vitro. With further investigation we may decipher the mechanism of NKreg suppression and operationalize expansion of NKreg cells associated with cGvHD suppression.

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Cytomegalovirus-specific T cells restricted for shared and donor human leukocyte antigens differentially impact on Cytomegalovirus reactivation risk after allogeneichematopoietic stem cell transplantation.

We longitudinally monitored 75 consecutive patients for 1 year after allo-HSCT (n=630 samples). The presence of =0.5 CMV-specific CD8+ cells//L at day+45 was an independent protective factor from subsequent clinicallyrelevant reactivation in univariate(p.

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Development and manufacturing of novel locally produced anti-BCMA CART cells for the treatment of relapsed/refractory multiple myeloma: phase I clinical results.

Our findings demonstrate the manageable safety profile and efficacy of HBI0101. These favorable data are encouraging and support decentralization of CART production at an academic setting, ensuring a sufficient CART supply in the light of the increasing local demand.

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Heterogeneity in long term outcomes for R-ISS stage II in newly diagnosed multiple myeloma patients.

In conclusion, stratification of patients in the R-ISS stage II group can be improved by taking into account CA and ISS. However, this does not improve predictive performance of survival models.

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Impact of daratumumab based induction on stem cell collection parameters in Swedish myeloma patients.

Not available.

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Lenalidomide-based triplet regimens in first relapsed multiple myeloma patients: real-world evidence from a propensity score matched analysis.

The median progression-free survival (PFS) was significantly longer for DaraRd (29.8 vs 22.5 months; p=0.028). DaraRd was better tolerated, registering a lower rate of grade 3-4 non-hematological toxicity (OR: 0.4, p.

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Mutations in the RACGAP1 gene cause autosomal recessive congenital dyserythropoietic anemia type III.

Not available.

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One disease, many faces.

Not available.

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Oral HDAC inhibitor tucidinostat in patients with relapsed or refractory peripheral T-cell lymphoma: phase IIb results.

(ClinicalTrials. gov identifier: NCT02953652).

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Plasminogen activator-coated nanobubbles targeting cell-bound ß2-glycoprotein I as a novel thrombus-specific thrombolytic strategy.

Finally, treatment of C3-gain-of-function mice with rtPA-tNBs, that target ß2-GPI deposited in kidney glomeruli, decreased fibrin deposition, and improved urinalysis data with a greater efficiency than untargeted NBs. Our findings suggest that targeting cell-bound ß2-GPI may represent an efficient and thrombus-specific thrombolytic strategy in both APS-related and APSunrelated thrombotic conditions.

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Reply to Shao and Zhou.

Not available.

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The effects of cardio-selective beta blockade on diastolic dysfunction in children with sickle cell disease.

Not available.

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Thromb Haemost

Impact of Asymptomatic Superior Mesenteric Vein Thrombosis on the Outcomes of Patients with Liver Cirrhosis.

Asymptomatic SMV thrombosis may not influence the outcomes of cirrhotic patients. The timing of intervention for asymptomatic SMV thrombosis in liver cirrhosis should be further explored.

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Reviews&Editorials

Plenty of the editorials are available as full text through the publisher website using the provided link

Am J Hematol

Antibody drug conjugates for the treatment of Multiple Myeloma.

Strategies to mitigate ocular events for Belantamab Mafodotin involve lower and less frequent dosing as well as the use of gamma secretase inhibitors. The optimal sequencing of ADCs within the treatment pathway including novel immunotherapies is now under evaluation.

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The immune system in multiple myeloma and precursor states: lessons and implications for immunotherapy and interception.

Optimal application and sequencing of these new immune therapies and their integration into clinical MM management may depend on the underlying immune status, in turn impacted by host, tumor and environmental features. Immune therapies carry the potential to achieve durable unmaintained responses and cures in MM.

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Trial designs and endpoints for immune therapies in multiple myeloma.

We then highlight trial designs to optimize the selection of dose and schedule, explore rational combination therapies based on preclinical data, and evaluate the nuances of commonly used endpoints. By exploiting their pharmacokinetic/pharmacodynamic profiles and utilizing novel translational insights, we can optimize the use of immune therapies in multiple myeloma.

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Ann Oncol

Precision Oncology for Biliary Tract Tumors: It's Written in Blood!

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Blood

Do you need the immune system to cure ALL?

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Endothelial cell-mediated venous thrombosis.

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Lost in translation: cytoplasmic UBA1 and VEXAS syndrome.

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Raise the roof, build the floor.

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Blood Cancer J

Does the pursuit of scientific excellence serve or hamper translational medical research: an historical perspective from hematological malignancies.

We then discuss the history of arsenic trioxide as additional APL therapy, and the repurposing of thalidomide as effective multiple myeloma therapy. These stories have surprising elements of commonality that demand debate about the modern-day hard and soft governance of medical research and whether these processes appropriately align the priorities of advancing scientific knowledge and the need of patients.

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J Hematol Oncol

BTK inhibitors in the treatment of hematological malignancies and inflammatory diseases: mechanisms and clinical studies.

Promising data have been obtained from preclinical and early-phase clinical studies. In this review, we summarized current progress in applying BTK inhibitors in the treatment of hematological malignancies and inflammatory disorders, highlighting available results from clinical studies.

Pubmed   Journal   ReadQx   PMC

Lancet Haematol

Access to and affordability of CAR T-cell therapy in multiple myeloma: an EBMT position paper.

Here, we identify two important issues: affordability and access to CAR T-cell treatment. This consensus statement from clinical investigators, clinicians, nurses, and patients from the European Society for Blood and Marrow Transplantation (EBMT) proposes solutions as part of an innovative collaborative strategy to make CAR T-cell therapy accessible to al patients with multiple myeloma.

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Caregivers-an undervalued and underused essential.

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Faecal microbiota transplantation in patients with haematological malignancies undergoing cellular therapies: from translational research to routine clinical practice.

Therefore, faecal microbiota transplantation has been evaluated in patients with haematological malignancies for various indications, including Clostridioides difficile infection, eradication of multidrug-resistant bacteria, and steroid refractory acute GVHD. In addition, use of prophylactic faecal microbiota transplantation to restore the gut microbiota and improve patients' outcomes is being developed in the setting of allogeneic HCT, but also probably very soon in patients receiving autologous HCT or CAR T cells.

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The influence of drug prices, new availability of inexpensive generic imatinib, new approvals, and post-marketing research on the treatment of chronic myeloid leukaemia in the USA.

The role of third-generation TKIs as second-line therapy following front-line resistance to second-generation TKIs needs to be evaluated. New and mature data with TKI therapy in chronic myeloid leukaemia are producing observations that encourage continuous discussion of the optimal treatment recommendations and frameworks in chronic myeloid leukaemia.

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Miscellaneous

misc publications eg case reports, tools of the trade, images of the month, etc…

Am J Hematol

In Memoriam: Dr. Carlton Haywood, Jr. (January 26, 1976 - December 31, 2021).

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Blood

An unusual myelofibrosis.

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Blastic plasmacytoid dendritic cell neoplasm in ocular adnexa.

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Description of a novel subtype of acute myeloid leukemia defined by recurrent CBFB insertions.

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Freeman SD, Valk P. Transplant in older adults with AML: genomic wheat and chaff. Blood. 2022;139(24):3459-3461.

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HSCT for adults with immunodeficiencies.

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Lithium attenuates graft-versus-host disease via effects on the intestinal stem cell niche.

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STAT1 and MHCII: guardians of stressed HSCs.

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Steiner L. Helping GATA1 make complex decisions. Blood. 2022;139(24):3457-3459.

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TLR1/2-stimulated DCs "prime" HSCs via IL-1ß.

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Two's company but three's a blast!

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Blood Adv

Gender equity analysis of nontrainee hemostasis and thrombosis recognition award recipients.

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Haematologica

How we changed our approach to venous thromboembolism.

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Images from the Haematologica Atlas of Hematologic Cytology: myelodysplastic syndrome with isolated del(5q).

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Lancet Haematol

A survey on the patient perspective on cure in multiple myeloma.

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Monika Asnani - addressing the unmet needs of sickle cell disease.

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Should patients with haemophilia receive gene therapy?

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The underrepresented majority.

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Venetoclax activity in a patient with central nervous system involvement by chronic lymphocytic leukaemia.

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Leukemia

Luspatercept mitigates bone loss driven by myelodysplastic neoplasms and estrogen-deficiency in mice.

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Organ dysfunction, thrombotic events and malignancies in patients with idiopathic multicentric castleman disease: a population-level US health claims analysis.

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Letters&Replies

Letters to the editors and authors’ replies

Am J Hematol

Limited activity of luspatercept in myelofibrosis and myeloid neoplasms other than myelodysplastic syndromes with ring sideroblasts.

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Subsequent anti-myeloma therapy after maturation antigen (BCMA) chimeric antigen receptor (CAR)-T cell (HDS269B) treatment in patients with relapsed/refractory multiple myeloma.

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Blood Cancer J

Fatal Powassan virus encephalitis in patients with chronic lymphocytic leukemia.

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TET2 deficiency promotes MDS-associated leukemogenesis.

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J Hematol Oncol

Circulating tumor DNA integrating tissue clonality detects minimal residual disease in resectable non-small-cell lung cancer.

Longitudinal ctDNA surveillance integrating clonality information may stratify high-risk patients with disease recurrence and infer the evolutionary origin of ctDNA mutations.

Pubmed   Journal   ReadQx   PMC

Gene and cell therapies in China: booming landscape under dual-track regulation.

These data showed that current dual regulation tracks in China complemented each other and together facilitated the GCT development, especially after 2017. More consistent technical standards and risk-based regulation will help bring more GCT products to patients.

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Leukemia

The WHO classification of haematolymphoid tumours: response to Swerdlow et al.

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